Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States.
“We recognize the considerable needs facing the DMD44 community given there are no approved exon skipping therapies for this disease as well as the potential of del-zota based on the unprecedented data shown in our clinical studies,” said Sarah Boyce, President and Chief Executive Officer of Avidity. “We are pleased to open this MAP to enable a compliant approach to providing del-zota to eligible patients as quickly as possible.”
Under an FDA-authorized treatment protocol, Avidity will provide del-zota to eligible boys and men with DMD44 through participating healthcare providers. Enrollment is anticipated to begin by year end, and participants in EXPLORE44-OLE will have the option to transition to the MAP as they complete 2 years of treatment.
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Avidity aligned with FDA on a path forward for a BLA submission for del-zota following an October 2025 pre-BLA meeting, with the submission planned for 2026 for accelerated approval. Participants will transition to commercial drug supply upon future potential FDA approval and product availability.
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